PE20212267A1 - NOVEL AAV CAPSIDES AND COMPOSITIONS CONTAINING THEM - Google Patents
NOVEL AAV CAPSIDES AND COMPOSITIONS CONTAINING THEMInfo
- Publication number
- PE20212267A1 PE20212267A1 PE2021001795A PE2021001795A PE20212267A1 PE 20212267 A1 PE20212267 A1 PE 20212267A1 PE 2021001795 A PE2021001795 A PE 2021001795A PE 2021001795 A PE2021001795 A PE 2021001795A PE 20212267 A1 PE20212267 A1 PE 20212267A1
- Authority
- PE
- Peru
- Prior art keywords
- novel aav
- compositions containing
- capsides
- aav capsides
- novel
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title 1
- 210000000234 capsid Anatomy 0.000 abstract 2
- 230000026683 transduction Effects 0.000 abstract 1
- 238000010361 transduction Methods 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14151—Methods of production or purification of viral material
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
Abstract
En la presente se proporcionan nuevas capsides de AAV y rAAV que las comprenden. En una forma de realizacion, los vectores que utilizan una nueva capside de AAV demuestran una transduccion mayor de un tejido diana seleccionado en comparacion con un AAV del arte previoNovel AAV and rAAV capsids comprising them are provided herein. In one embodiment, vectors utilizing a novel AAV capsid demonstrate greater transduction of a selected target tissue compared to a prior art AAV.
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962840184P | 2019-04-29 | 2019-04-29 | |
US201962913314P | 2019-10-10 | 2019-10-10 | |
US201962924095P | 2019-10-21 | 2019-10-21 | |
PCT/US2020/030281 WO2020223236A1 (en) | 2019-04-29 | 2020-04-28 | Novel aav capsids and compositions containing same |
Publications (1)
Publication Number | Publication Date |
---|---|
PE20212267A1 true PE20212267A1 (en) | 2021-11-30 |
Family
ID=73028724
Family Applications (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PE2021001788A PE20220014A1 (en) | 2019-04-29 | 2020-04-28 | NOVEL AAV CAPSIDES AND COMPOSITIONS CONTAINING THEM |
PE2021001789A PE20212334A1 (en) | 2019-04-29 | 2020-04-28 | NOVEL AAV CAPSIDES AND COMPOSITIONS CONTAINING THEM |
PE2021001795A PE20212267A1 (en) | 2019-04-29 | 2020-04-28 | NOVEL AAV CAPSIDES AND COMPOSITIONS CONTAINING THEM |
Family Applications Before (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PE2021001788A PE20220014A1 (en) | 2019-04-29 | 2020-04-28 | NOVEL AAV CAPSIDES AND COMPOSITIONS CONTAINING THEM |
PE2021001789A PE20212334A1 (en) | 2019-04-29 | 2020-04-28 | NOVEL AAV CAPSIDES AND COMPOSITIONS CONTAINING THEM |
Country Status (15)
Country | Link |
---|---|
US (3) | US20220220453A1 (en) |
EP (3) | EP3976078A4 (en) |
JP (3) | JP2022530544A (en) |
KR (3) | KR20220004680A (en) |
CN (3) | CN113853208A (en) |
AU (3) | AU2020266523A1 (en) |
BR (2) | BR112021020545A2 (en) |
CA (3) | CA3134379A1 (en) |
CL (3) | CL2021002768A1 (en) |
CO (3) | CO2021015924A2 (en) |
IL (3) | IL287492A (en) |
MX (3) | MX2021013266A (en) |
PE (3) | PE20220014A1 (en) |
SG (3) | SG11202111375RA (en) |
WO (3) | WO2020223232A1 (en) |
Families Citing this family (13)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
IL299167A (en) | 2020-06-17 | 2023-02-01 | Univ Pennsylvania | Compositions and methods for treatment of gene therapy patients |
US20230372539A1 (en) | 2020-08-24 | 2023-11-23 | The Trustees Of The University Of Pennsylvania | Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases |
WO2022094078A1 (en) | 2020-10-28 | 2022-05-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
TW202233584A (en) | 2020-11-11 | 2022-09-01 | 歐洲分子生物學實驗室 | Modified viral particles for gene therapy |
TW202237850A (en) | 2020-12-01 | 2022-10-01 | 賓州大學委員會 | Novel compositions with tissue-specific targeting motifs and compositions containing same |
IL303239A (en) * | 2020-12-01 | 2023-07-01 | Univ Pennsylvania | Compositions and uses thereof for treatment of angelman syndrome |
AU2022262771A1 (en) | 2021-04-23 | 2023-11-02 | The Trustees Of The University Of Pennsylvania | Novel compositions with brain-specific targeting motifs and compositions containing same |
WO2023147304A1 (en) | 2022-01-25 | 2023-08-03 | The Trustees Of The University Of Pennsylvania | Aav capsids for improved heart transduction and detargeting of liver |
WO2023168293A2 (en) * | 2022-03-01 | 2023-09-07 | The Trustees Of The University Of Pennsylvania | Viral vector genome encoding an insulin fusion protein |
WO2023168403A2 (en) * | 2022-03-03 | 2023-09-07 | The Trustees Of The University Of Pennsylvania | Viral vectors encoding parathyroid hormone fusions and uses thereof in treating hypoparathyroidism |
WO2023196893A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating her2 positive metastatic breast cancer and other cancers |
WO2023196892A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
WO2024015966A2 (en) * | 2022-07-15 | 2024-01-18 | The Trustees Of The University Of Pennsylvania | Recombinant aav having aav clade d and clade e capsids and compositions containing same |
Family Cites Families (18)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
ES2258601T3 (en) * | 2001-11-13 | 2006-09-01 | The Trustees Of The University Of Pennsylvania | A METHOD FOR THE IDENTIFICATION OF THE UNKNOWN SEQUENCES OF ADENO-ASSOCIATED VIRUSES (VAA) AND A KIT FOR THE METHOD. |
CN1856576B (en) * | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
EP2359865B1 (en) * | 2005-04-07 | 2013-10-02 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
WO2008027084A2 (en) * | 2006-04-28 | 2008-03-06 | The Trustees Of The University Of Pennsylvania | Modified aav vectors having reduced capsid immunogenicity and use thereof |
WO2011133890A1 (en) * | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
KR102572449B1 (en) * | 2014-03-10 | 2023-08-31 | 유니큐어 아이피 비.브이. | Further improved aav vectors produced in insect cells |
WO2015191508A1 (en) * | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
EP3632923A1 (en) * | 2015-01-16 | 2020-04-08 | Voyager Therapeutics, Inc. | Central nervous system targeting polynucleotides |
AU2016298394C1 (en) * | 2015-07-30 | 2024-02-15 | Massachusetts Eye And Ear Infirmary | Ancestral virus sequences and uses thereof |
US20180230489A1 (en) * | 2015-10-28 | 2018-08-16 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
EP3368065A4 (en) * | 2015-10-29 | 2019-03-20 | Voyager Therapeutics, Inc. | Delivery of central nervous system targeting polynucleotides |
EP3448874A4 (en) * | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
EP3458588A4 (en) * | 2016-05-18 | 2020-01-15 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
AU2017345470B2 (en) * | 2016-10-19 | 2023-08-03 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
US10550405B2 (en) * | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
WO2018200419A1 (en) * | 2017-04-23 | 2018-11-01 | The Trustees Of The University Of Pennsylvania | Viral vectors comprising engineered aav capsids and compositions containing the same |
JOP20190269A1 (en) * | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
EP3808849A1 (en) * | 2017-08-03 | 2021-04-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
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2020
- 2020-04-28 US US17/605,874 patent/US20220220453A1/en active Pending
- 2020-04-28 WO PCT/US2020/030273 patent/WO2020223232A1/en active Application Filing
- 2020-04-28 SG SG11202111375RA patent/SG11202111375RA/en unknown
- 2020-04-28 EP EP20798752.0A patent/EP3976078A4/en active Pending
- 2020-04-28 CN CN202080032773.1A patent/CN113853208A/en active Pending
- 2020-04-28 WO PCT/US2020/030266 patent/WO2020223231A1/en active Application Filing
- 2020-04-28 BR BR112021020545A patent/BR112021020545A2/en unknown
- 2020-04-28 JP JP2021564542A patent/JP2022530544A/en active Pending
- 2020-04-28 MX MX2021013266A patent/MX2021013266A/en unknown
- 2020-04-28 JP JP2021564269A patent/JP2022530633A/en active Pending
- 2020-04-28 MX MX2021013268A patent/MX2021013268A/en unknown
- 2020-04-28 US US17/605,881 patent/US20220249705A1/en active Pending
- 2020-04-28 AU AU2020266523A patent/AU2020266523A1/en active Pending
- 2020-04-28 CA CA3134379A patent/CA3134379A1/en active Pending
- 2020-04-28 CN CN202080033196.8A patent/CN113853209A/en active Pending
- 2020-04-28 WO PCT/US2020/030281 patent/WO2020223236A1/en active Application Filing
- 2020-04-28 SG SG11202111373YA patent/SG11202111373YA/en unknown
- 2020-04-28 EP EP20798753.8A patent/EP3962507A4/en active Pending
- 2020-04-28 KR KR1020217037659A patent/KR20220004680A/en unknown
- 2020-04-28 AU AU2020264976A patent/AU2020264976A1/en active Pending
- 2020-04-28 CA CA3134507A patent/CA3134507A1/en active Pending
- 2020-04-28 MX MX2021013267A patent/MX2021013267A/en unknown
- 2020-04-28 BR BR112021021502A patent/BR112021021502A2/en unknown
- 2020-04-28 CN CN202080032666.9A patent/CN113853207A/en active Pending
- 2020-04-28 PE PE2021001788A patent/PE20220014A1/en unknown
- 2020-04-28 JP JP2021564543A patent/JP2022530656A/en active Pending
- 2020-04-28 EP EP20798442.8A patent/EP3976077A4/en active Pending
- 2020-04-28 US US17/605,868 patent/US20220204990A1/en active Pending
- 2020-04-28 CA CA3134468A patent/CA3134468A1/en active Pending
- 2020-04-28 PE PE2021001789A patent/PE20212334A1/en unknown
- 2020-04-28 SG SG11202111102SA patent/SG11202111102SA/en unknown
- 2020-04-28 KR KR1020217038207A patent/KR20220004695A/en unknown
- 2020-04-28 AU AU2020264975A patent/AU2020264975A1/en active Pending
- 2020-04-28 PE PE2021001795A patent/PE20212267A1/en unknown
- 2020-04-28 KR KR1020217037661A patent/KR20220004681A/en unknown
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2021
- 2021-10-21 IL IL287492A patent/IL287492A/en unknown
- 2021-10-21 CL CL2021002768A patent/CL2021002768A1/en unknown
- 2021-10-21 IL IL287491A patent/IL287491A/en unknown
- 2021-10-21 IL IL287498A patent/IL287498A/en unknown
- 2021-10-26 CL CL2021002811A patent/CL2021002811A1/en unknown
- 2021-10-27 CL CL2021002824A patent/CL2021002824A1/en unknown
- 2021-11-25 CO CONC2021/0015924A patent/CO2021015924A2/en unknown
- 2021-11-25 CO CONC2021/0015916A patent/CO2021015916A2/en unknown
- 2021-11-25 CO CONC2021/0015928A patent/CO2021015928A2/en unknown
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